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Pfizer and BioNTech to Provide 200 Million Doses of COVID-19 Vaccine for U.S.

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Pfizer and BioNTech to Provide 200 Million Doses of COVID-19 Vaccine for U.S.

 July 23, 2021: “Pfizer and BioNTech announced that the U.S. government has purchased an additional 200 million doses of the Pfizer-BioNTech COVID-19 Vaccine.

These doses are expected to be delivered from October 2021 through April 2022.

This brings the total number of doses to be supplied by the companies to the U.S. government under its existing supply agreement to 500 million.

In a separate announcement on June 10, 2021, Pfizer and BioNTech shared plans to provide the U.S. government with 500 million doses of the companies’ COVID-19 vaccine for donation to the world’s poorest nations.

“As a long-term partner to the U.S. government in the fight against this pandemic, we are proud of the impact of vaccination efforts across the country.

Vaccines have been and will remain critical to protecting lives against this devastating disease,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer.

“These additional doses will help the U.S. government ensure broad vaccine access into next year.”

“It has been our consistent goal to supply as many doses of our COVID-19 vaccine as possible to people around the world to help bring an end to this pandemic,” said Ugur Sahin, M.D., CEO and Co-founder of BioNTech.

“We are honored to support the U.S. and more than 100 countries in their continuing vaccination programs.”

The companies expect to deliver 110 million of the additional doses by December 31, 2021, with the remaining 90 million doses to be delivered no later than April 30, 2022.

The U.S. government also has the option to acquire an updated version of the vaccine to address potential variants as well as new formulations of the vaccine, if available and authorized.

Eligible U.S. residents will continue to receive the vaccine for free, consistent with the U.S. government’s commitment for free access to COVID-19 vaccines.

The Pfizer-BioNTech COVID-19 Vaccine, which is based on BioNTech proprietary mRNA technology, was developed by both BioNTech and Pfizer. BioNTech is the Marketing Authorization Holder in the European Union, and the holder of emergency use authorizations or equivalent in the United States (jointly with Pfizer), Canada and other countries in advance of a planned application for full marketing authorizations in these countries.

The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. FDA, but has been authorized for emergency use by FDA under an EUA to prevent Coronavirus Disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) for use in individuals 12 years of age and older.

The emergency use of this product is only authorized for the duration of the declaration that circumstances exist justifying the authorization of emergency use of the medical product under Section 564 (b) (1) of the FD&C Act unless the declaration is terminated or authorization revoked sooner.

Please see Emergency Use Authorization (EUA) Fact Sheet for Healthcare Providers Administering Vaccine (Vaccination Providers) and Full EUA Prescribing Information available at www.cvdvaccine-us.com.

AUTHORIZED USE IN THE U.S.:

The Pfizer-BioNTech COVID-19 Vaccine is authorized for use under an Emergency Use Authorization (EUA) for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 12 years of age and older.

IMPORTANT SAFETY INFORMATION FROM U.S. FDA EMERGENCY USE AUTHORIZATION PRESCRIBING INFORMATION:

  • Do not administer Pfizer-BioNTech COVID-19 Vaccine to individuals with known history of a severe allergic reaction (e.g., anaphylaxis) to any component of the Pfizer-BioNTech COVID-19 Vaccine
  • Appropriate medical treatment used to manage immediate allergic reactions must be immediately available in the event an acute anaphylactic reaction occurs following administration of Pfizer-BioNTech COVID-19 Vaccine

    Monitor Pfizer-BioNTech COVID-19 Vaccine recipients for the occurrence of immediate adverse reactions according to the Centers for Disease Control and Prevention guidelines (https://www.cdc.gov/vaccines/covid-19/clinical-considerations/managing-anaphylaxis.html)
  • Reports of adverse events following use of the Pfizer-BioNTech COVID-19 Vaccine under EUA suggest increased risks of myocarditis and pericarditis, particularly following the second dose.

    The decision to administer the Pfizer-BioNTech COVID-19 Vaccine to an individual with a history of myocarditis or pericarditis should take into account the individual’s clinical circumstances
  • Syncope (fainting) may occur in association with administration of injectable vaccines, in particular in adolescents. Procedures should be in place to avoid injury from fainting
  • Immunocompromised persons, including individuals receiving immunosuppressant therapy, may have a diminished immune response to the Pfizer-BioNTech COVID-19 Vaccine
  • The Pfizer-BioNTech COVID-19 Vaccine may not protect all vaccine recipients
  • In clinical studies, adverse reactions in participants 16 years of age and older included pain at the injection site (84.1%), fatigue (62.9%), headache (55.1%), muscle pain (38.3%), chills (31.9%), joint pain (23.6%), fever (14.2%), injection site swelling (10.5%), injection site redness (9.5%), nausea (1.1%), malaise (0.5%), and lymphadenopathy (0.3%)
  • In a clinical study, adverse reactions in adolescents 12 through 15 years of age included pain at the injection site (90.5%), fatigue (77.5%), headache (75.5%), chills (49.2%), muscle pain (42.2%), fever (24.3%), joint pain (20.2%), injection site swelling (9.2%), injection site redness (8.6%), lymphadenopathy (0.8%), and nausea (0.4%)
  • Following administration of the Pfizer-BioNTech COVID-19 Vaccine, the following have been reported outside of clinical trials:
    • severe allergic reactions, including anaphylaxis, and other hypersensitivity reactions, diarrhea, vomiting, and pain in extremity (arm)
    • myocarditis and pericarditis
      Additional adverse reactions, some of which may be serious, may become apparent with more widespread use of the Pfizer-BioNTech COVID-19 Vaccine
  • Available data on Pfizer-BioNTech COVID-19 Vaccine administered to pregnant women are insufficient to inform vaccine-associated risks in pregnancy
  • Data are not available to assess the effects of Pfizer-BioNTech COVID-19 Vaccine on the breastfed infant or on milk production/excretion
  • There are no data available on the interchangeability of the Pfizer-BioNTech COVID-19 Vaccine with other COVID-19 vaccines to complete the vaccination series.

    Individuals who have received one dose of Pfizer-BioNTech COVID-19 Vaccine should receive a second dose of Pfizer-BioNTech COVID-19 Vaccine to complete the vaccination series
  • Vaccination providers must report Adverse Events in accordance with the Fact Sheet to VAERS online at https://vaers.hhs.gov/reportevent.html.

    For further assistance with reporting to VAERS call 1-800-822-7967. The reports should include the words “Pfizer-BioNTech COVID-19 Vaccine EUA” in the description section of the report
  • Vaccination providers should review the Fact Sheet for Information to Provide to Vaccine Recipients/Caregivers and Mandatory Requirements for Pfizer-BioNTech COVID-19 Vaccine Administration Under Emergency Use Authorization
  • Before administration of Pfizer-BioNTech COVID-19 Vaccine, please see Emergency Use Authorization (EUA) Fact Sheet for Healthcare Providers Administering Vaccine (Vaccination Providers) including Full EUA Prescribing Information available at www.cvdvaccine-us.com”


    https://www.pfizer.com/news/press-release/press-release-detail/pfizer-and-biontech-provide-us-government-additional-200

Alexion’s Ultomiris (ravulizumab) recommended for approval in EU for PNH

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Alexion’s Ultomiris (ravulizumab) recommended for approval in EU for PNH

July 26, 2021: Alexion’s Ultomiris (ravulizumab) has been recommended for marketing authorisation in the EU for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on interim results from the Phase III clinical trial in children and adolescents with PNH, which were recently presented during the European Hematology Association 2021 Virtual Congress.

This trial demonstrated that Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks for the treatment of children and adolescents up to 18 years of age with PNH.

Additionally, Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough haemolysis, which can lead to disabling or potentially fatal blood clots.

The efficacy and safety of Ultomiris in children and adolescents is consistent with the established profile of Ultomiris in clinical trials involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting.2,3

PNH is an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis (blood clots), which can occur throughout the body, and result in organ damage and potentially premature death.1,4-6

Austin Kulasekararaj, MD, King’s College Hospital, London, United Kingdom, said: “PNH can have a significant and devastating impact on a child’s quality of life, and it can be overwhelming for families to manage their disease.

The potential approval of Ultomiris, which offers the efficacy and safety already established with Soliris (eculizumab)and requires fewer treatments each year, would have a meaningful impact for children with PNH and their families.”

Marc Dunoyer, incoming Chief Executive Officer, Alexion, said: “This recommendation shows that Ultomiris – which has become the standard of care for the treatment of adults with PNH – has the potential to transform the lives of children and adolescents in Europe suffering from this devastating rare disease.

As we listen to the patient community and understand the challenges of living with a rare disease, we recognize the importance of continuing to deliver options and formulations that enhance patient care and disease management.”

The CHMP recommended the expanded use of Ultomiris to include children (with a body weight of 10 kg or above) and adolescents with PNH who experience haemolysis with clinical symptom(s) indicative of high disease activity, as well as for individuals who are clinically stable after having been treated with Solirisfor at least the past six months.

Ultomiris was first approved in the EU in 2019 for the treatment of adults with PNH and is also approved in the EU for the treatment of adults and children with atypical haemolytic uraemic syndrome (aHUS).

In June 2021, the US FDA approved the expanded use of Ultomiris to include children (one month of age and older) and adolescents with PNH, the first and only treatment for this age group in the US.

PNH
Paroxysmal nocturnal haemoglobinuria (PNH) is a serious ultra-rare blood disorder with devastating consequences.

It is characterised by the destruction of red blood cells, which is also referred to as haemolysis. PNH occurs when the complement system – a part of the body’s immune system – over-responds, leading the body to attack its own red blood cells.

PNH often goes unrecognised, with delays in diagnosis from one to more than five years. Patients with PNH may experience a range of symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-coloured urine and anaemia.

The most devastating consequence of chronic haemolysis is the formation of blood clots, which can occur in blood vessels throughout the body, damage vital organs, and potentially lead to premature death.

The prognosis of PNH can be poor in many cases, so a timely and accurate diagnosis – in addition to appropriate treatment – is critical to improving patient outcomes.

Ultomiris
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, offers immediate, complete, and sustained complement inhibition.

The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system.

When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks or, for paediatric patients less than 20 kg, every four weeks, following a loading dose.

 Ultomiris is approved in the US for the treatment of adults and children (one month of age and older) with PNH, as well as in the EU and Japan as a treatment for adults with PNH.

It is also approved in the US for aHUS to inhibit complement-mediated thrombotic microangiopathy in adult and paediatric (one month of age and older) patients, in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS, as well as in Japan for adults and children with aHUS.

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/ultomiris-recommended-in-eu-for-children-with-pnh.html

AZ’s Vaxzevria is highly effective after one dose for Beta and Delta variants

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AZ’s Vaxzevria is highly effective after one dose for Beta and Delta variants

July 23,2021: “Results from the Canadian Immunization Research Network (CIRN) with support from Public Health Agency of Canada and the Canadian Institutes of Health Research, published as a pre-print, demonstrated one dose of Vaxzevria was 82% effective against hospitalisation or death caused by the Beta/Gamma variants of the SARS-CoV-2 virus.

The vaccine also showed a high level of effectiveness against the Delta (B.617.2, ‘Indian’) and Alpha (B.1.1.7, ‘Kent’) variants with an 87% and 90% reduction of hospitalisations or deaths respectively.

The effectiveness of Vaxzevria after one dose against hospitalisation or death was similar to that of other vaccines tested in the study.

Follow-up time was not sufficient to report on effectiveness of Vaxzevria after two doses, other studies have shown an increased effectiveness following the indicated two dose schedule.

Vaxzevria was effective against milder symptomatic disease although, given that data was only reported after a first dose instead of the indicated two dose schedule where efficacy is known to be enhanced in this disease setting,2 the efficacy was lower than against severe disease. Vaccine effectiveness against any symptomatic disease was 50% against the Beta/Gamma variants, and 70% and 72% against the Delta and Alpha variants, respectively.

A Phase I/II trial carried out by the University of Oxford and University of the Witwatersrand in January, had shown limited efficacy against mild disease primarily due to the Beta variant. It was unable to properly ascertain vaccine efficacy against severe disease including  hospitalisation and death given that subjects were predominantly young, healthy adults who experienced mild disease only.3

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, said: “With different variants threatening to disrupt our route out of the pandemic, this real-world evidence shows that Vaxzevria, along with other vaccines used in Canada, provides a high level of protection against the most serious forms of the disease, even after just one shot. It is essential that we continue to protect as many people as possible in all corners of the world in order to get ahead of this deadly virus.”

The analysis included 69,533 individuals who tested positive for SARS-CoV-2 during December 2020 to May 2021 in Ontario, Canada; with 28,705 (6.8%) positive for non-variants of concern SARS-CoV-2 and 40,828 (9.7%) positive for a variant of concern.

Vaxzevria, formerly AZD1222
Vaxzevria was co-invented by the University of Oxford and its spin-out company, Vaccitech. It uses a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 virus spike protein. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.

The vaccine has been granted a conditional marketing authorisation or emergency use in more than 80 countries across six continents. 

More than 700 million doses of COVID-19 Vaccine AstraZeneca have been supplied to 170 countries worldwide, including more than 100 countries through the COVAX Facility.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/vaxzevria-is-highly-effective-after-one-dose-against-severe-disease-or-hospitalisation-caused-by-beta-and-delta-variants-of-concern.html

Sanofi continues streamlining of established products with sale of dental care brands to Septodont

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Sanofi continues streamlining of established products with sale of dental care brands to Septodont

July 22 2021: “Sanofi has concluded an agreement to divest an integrated portfolio of dental care brands and related medical devices to Septodont, a French privately-owned company headquartered near Paris.

Divested portfolio includes four dental care brands Ultracain®, Rodogyl®, Birodogyl® and Dontisolon®, as well as related medical devices, mostly marketed in Europe.

This portfolio of high-quality medicines benefits patients both during and after dental operations, while covering most dental indications (anesthetics, anti-infective and anti-inflammatory).

This transaction continues Sanofi’s ongoing strategic transformation announced in December 2019.

The agreement covers the registrations, trademarks, and related commercial rights. This is an asset deal. No employees, equipment or real estate is included.
Commercial terms of the agreement will not be disclosed.

About Septodont

Septodont is a leading manufacturer of dental pharmaceuticals and medical devices.

Since its inception in 1932, the group develops, produces and distributes a large range of products and solutions dedicated to serving the needs of dental professionals across the world.

The group counts 1,900 employees and has a long-established presence in more than 150 countries.

Beyond dental and through its Novocol Pharma division, the group also partners with pharmaceutical and medical device companies to provide contract manufacturing services for pharmaceutical products that require cartridge filling.

Learn more about Septodont’s innovative, safe and healthcare solutions at https://www.septodontcorp.com/.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-07-22-14-00-00-2267219

Myovant Sciences Announces EU Approval for RYEQO® for Uterine Fibroids

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Myovant Sciences Announces EU Approval for RYEQO® for Uterine Fibroids

July 20, 2021: “Myovant Sciences announced the European Commission (EC) has approved the marketing authorization application for RYEQO® (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age, with no limitation for duration of use. 

The EC decision is valid in all 27 member states of the European Union, as well as Iceland, Norway, and Liechtenstein.

“Data from the Phase 3 LIBERTY program, which supported the approval of RYEQO, showed that RYEQO improved symptoms most relevant to women living with uterine fibroids, namely heavy menstrual bleeding and pain, while maintaining a well-tolerated safety profile,” said Roberta Venturella, M.D., Ph.D., Associate Professor, Magna Græcia University of Catanzaro and investigator in the LIBERTY program.

“With this approval, women and doctors finally have a long-term treatment option, which is important for the management of this condition.”

“Today’s approval of RYEQO, the first and only once-daily long-term treatment for women with uterine fibroids in Europe, marks a major milestone in expanding non-invasive treatment options for this common and potentially debilitating disease,” said David Marek, Chief Executive Officer of Myovant Sciences, Inc.

“Through our partnership with Gedeon Richter, we look forward to supporting even more women suffering from uterine fibroids.”

The approval is based on safety and efficacy data from the Phase 3 LIBERTY program, which consisted of two replicate, 24-week, multinational clinical studies (LIBERTY 1 and LIBERTY 2), a one-year extension study, and supportive bone mineral density data from a randomized withdrawal study.

Results from the LIBERTY 1 and LIBERTY 2 studies were published in the New England Journal of Medicine in February 2021.

In March 2020, Myovant and Gedeon Richter entered into an exclusive license agreement for Gedeon Richter to commercialize relugolix combination tablet for uterine fibroids and endometriosis in Europe, the Commonwealth of Independent States including Russia, Latin America, Australia, and New Zealand.

Under the agreement, Myovant received an upfront payment of $40 million and is eligible to receive up to $40 million in regulatory milestones and $107.5 million in sales milestones for a total of $147.5 million, and tiered royalties on net sales following regulatory approval. 

Gedeon Richter will be responsible for local clinical development, manufacturing, and all commercialization for its territories. 

Myovant and Gedeon Richter will also continue to collaborate on the marketing authorization application for endometriosis, which is expected to be submitted in the second half of calendar year 2021.

About Uterine Fibroids
Uterine fibroids are noncancerous tumors that develop in or on the muscular walls of the uterus and are among the most common reproductive tract tumors in women.

In addition to an individual’s genetic predisposition, estrogens are well known to play an important role in the regulation of fibroid growth.

Although uterine fibroids are benign tumors, they can cause debilitating symptoms such as heavy menstrual bleeding (frequently resulting in anemia and fatigue), pain (including painful periods, abdominal pain, painful intercourse, backache), increased abdominal girth and bloating, urinary frequency or retention, constipation, pregnancy loss, and, in some cases, infertility.

These symptoms can also lead to loss of productivity at work, limitations in normal activities of daily living, and social embarrassment.

About RYEQO®
RYEQO (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is approved for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age.

RYEQO contains relugolix, which reduces the amount of estrogen (and other hormones) produced by ovaries, estradiol (an estrogen), which may reduce the risk of bone loss, and norethindrone acetate (a progestin), which is necessary when women with a uterus (womb) take estrogen.”

https://investors.myovant.com/news-releases/news-release-details/myovant-sciences-announces-european-commission-approval-ryeqor

Bayer’s new symptomatic chronic heart failure treatment Verquvo™

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Bayer’s new symptomatic chronic heart failure treatment Verquvo™

July 21, 2021: “The European Commission has granted marketing authorization in the EU for vericiguat under the brand name Verquvo™.

Verquvo (2.5 mg, 5 mg, and 10 mg), a soluble guanylate cyclase (sGC) stimulator, is indicated for symptomatic chronic heart failure in adult patients with reduced ejection fraction who are stabilized after a recent decompensation event requiring intravenous (IV) therapy.

It works differently to existing heart failure treatments, providing a specific approach to managing chronic heart failure patients following a decompensation event, also known as a worsening event.

“With this latest approval, we have the potential to bring new hope to patients living with heart failure, by breaking the cycle of decompensation events, also known as worsening events, and reducing the risk of re-hospitalization,” said Dr. Burkert Pieske, professor of internal medicine and cardiology at Charité and principal investigator of the Phase III VICTORIA trial.

“Re-hospitalization has a significant impact on both patients and their families, and even when taking guideline-based therapy, many will still experience progressive worsening of symptoms.

Therefore, access to a new treatment that has been developed with these patients specifically in mind, is extremely welcome news.”

Current therapies block the harmful effects of the natural neurohormonal systems that are activated by the myocardial and vascular dysfunction present in heart failure. Vericiguat works in conjunction with existing approaches through a different mode of action.

It specifically restores the deficient NO-sGC-cGMP pathway, which plays a critical role in the progression of heart failure and aggravating its symptoms.

“The approval of Verquvo in the EU represents a significant breakthrough for those living with this condition,” said Dr. Michael Devoy, Chief Medical Officer and Head of Medical Affairs and Pharmacovigilance at Bayer’s Pharmaceuticals Division.

“As the leading cause of hospitalization in Europe, it is vital we continue to make such advances to improve patients’ quality of life. Currently half of patients with heart failure are readmitted within 30 days of hospitalization or initiation of intravenous diuretics.

We believe availability of Verquvo provides clinicians with a much-needed new option to help alleviate the huge burden of chronic heart failure.”

Verquvo (vericiguat) has been approved by the U.S. Food and Drug Administration (FDA) and the Ministry of Health, Labour, and Welfare (MHLW) in Japan.

Vericiguat has also been submitted for marketing authorization in China as well as multiple other countries worldwide.

Vericiguat is being jointly developed with MSD (a tradename of Merck & Co., Inc., Kenilworth, NJ, USA).

About Verquvo™ (vericiguat)
Verquvo 2.5 mg, 5 mg, and 10 mg is an oral once daily stimulator of soluble guanylate cyclase (sGC), an important enzyme in the nitric oxide (NO) signaling pathway.

When NO binds to sGC, the enzyme catalyzes the synthesis of intracellular cyclic guanosine monophosphate (cGMP), a second messenger that plays a role in the regulation of vascular tone, cardiac contractility, and cardiac remodeling.

Heart failure is associated with impaired synthesis of NO and decreased activity of sGC, which may contribute to myocardial and vascular dysfunction.

By directly stimulating sGC, independently of and synergistically with NO, vericiguat augments levels of intracellular cGMP, leading to smooth muscle relaxation and vasodilation.”

https://media.bayer.de/baynews/baynews.nsf/id/Bayers-new-symptomatic-chronic-heart-failure-treatment-Verquvo-vericiguat-approved-in-EU?OpenDocument&sessionID=1626944774

NANOPHARM LTD ACQUIRED BY APTARGROUP

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NANOPHARM LTD ACQUIRED BY APTARGROUP

June 11, 2019: “AptarGroup announced that it has acquired two leading pharmaceutical services companies, Nanopharm Ltd. and Gateway Analytical LLC, for a combined enterprise value of approximately $50 million.

The acquisitions are part of Aptar’s strategy to broaden its portfolio of services that support pharmaceutical and biotech customers to accelerate and derisk their complex product developments.

Both acquisitions bring complementary, value-added and differentiated analytical, testing and development services for all stages of drug development and commercialization.

The expanded services platform will enable Aptar Pharma to collaborate earlier with customers to support their complex drug formulations and delivery requirements as they face increasingly competitive and regulated markets.

Nanopharm, located in Newport, UK, is a science-driven, leading provider of orally inhaled and nasal drug product design and development services.

Gateway Analytical, located in Gibsonia, PA, provides industry-leading particulate detection and predictive analytical services to customers developing injectable medicines.

The companies’ results will be included in the Pharma segment and the transactions are not expected to be dilutive to Aptar’s earnings in 2019.

“We are delighted to welcome the talented teams of Nanopharm and Gateway and their customers to Aptar Pharma,” commented Gael Touya, President of Aptar Pharma.

“Their expertise and capabilities will deepen our current range of services offered to customers who are developing and testing inhaled and injected medicines.

This enables us to participate in a broader spectrum of the pre-clinical, clinical trial and market launch phases of drug development.”

Stephan Tanda, President and CEO, stated, “These strategic investments further strengthen our best in class Pharma business and increase our ability to add to our long-term growth pipeline by adding depth to our service offerings.

The expertise of two established leaders, Nanopharm, a leader in inhalation and nasal drug development services, and Gateway, a leader in particulate detection and analysis for the injectables market, are complementary to our existing laboratory and analytical services and will bring additional value to our customers as they navigate the challenging regulatory landscape and seek to bring their products to market faster.”

https://www.businesswire.com/news/home/20190610005180/en/Aptar-Acquires-Nanopharm-Gateway-Analytical-Broadening-Pharma

Albireo Announces FDA Approval of Bylvay,™ First Drug Treatment for Progressive Familial Intrahepatic Cholestasis

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Albireo Announces FDA Approval of Bylvay,™ First Drug Treatment for Progressive Familial Intrahepatic Cholestasis

July 20, 2021: “Albireo Pharma, a rare liver disease company developing novel bile acid modulators announced U.S. FDA approval of Bylvay (odevixibat), the first drug approved for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC).

Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi), which does not require refrigeration and is easily administered as a once-daily capsule or opened and sprinkled onto soft foods.

Albireo is launching Bylvay immediately to accelerate availability for the patients and families impacted by PFIC.

“Treating children with PFIC can be difficult and frustrating given the current treatment options.

Bylvay gives us a non-surgical option and will change how we treat PFIC,” said Richard Thompson, Professor of Molecular Hepatology at King’s College London and principal investigator of PEDFIC 1 and PEDFIC 2.

“With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed and we are hopeful for better outcomes for these children.”

PFIC is a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life.

The most prominent and problematic ongoing manifestation of PFIC is pruritus, or intense itching, which often results in a severely diminished quality of life.

Until now, there have been no approved drugs for PFIC. Only surgical options that include biliary diversion surgery (BDS) and liver transplantation have been available, and without them, most PFIC patients do not survive past the age of 30.

There are an estimated 100,000 patients with cholestatic liver disease without an approved drug treatment. Of those patients, there are approximately 15,000 with PFIC (excluding China and India).

“Until now invasive surgery was the only approved treatment option.

With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” said Emily Ventura, leader of PFIC Advocacy and Resource Network (www.pfic.org) and mother to a PFIC patient.

“As a community, we experience extreme challenges and diminished quality of life for children and families with PFIC. Managing the symptoms can be extremely difficult — the burden is unimaginable with our kids suffering physically, emotionally and developmentally.”

The approval of Bylvay was supported by data from PEDFIC 1 and PEDFIC 2, the largest, global, Phase 3 trials ever conducted in PFIC.

In PEDFIC 1, a randomized, double-blind, placebo-controlled study, Bylvay met both its pruritus (p=0.004) and serum bile acid (p=0.003) primary endpoints and was well tolerated with very low incidence of diarrhea/frequent bowel movements (9.5% of treated patients vs. 5.0% of placebo patients).

PEDFIC 2, a long-term, open-label Phase 3 extension study, reaffirmed Bylvay delivered sustained reductions in serum bile acids as well as improvements in pruritus assessments, growth and other markers of liver function in patients treated up to 48 weeks.

Across both studies, Bylvay was well tolerated with diarrhea/frequent stools being the most common treatment-related gastrointestinal adverse events.

There were no serious treatment-related adverse events.

“Bylvay is the first ever approval by the FDA of a drug developed for a pediatric cholestatic liver disease and provides a non-surgical treatment for patients living with the burden of PFIC,” said Ron Cooper, President and CEO of Albireo.

“We’re humbled by the children, families and investigators whose commitment to our clinical trials will bring hope and treatment benefit for so many future patients.”

Ready for Bylvay Launch
Bylvay is expected to be packaged and shipped within the coming days. With the immediate Bylvay launch, Albireo is ready with a focus on access and reimbursement, sales promotion, and patient support.

To support payor decision-making, Albireo is submitting a compelling value package with the PEDFIC, gold standard, Phase 3 data, which includes long-term data with patients on drug for over two years; natural history information; and a caregiver study to reflect the burden of PFIC.

Sales promotion will begin immediately.

The Albireo and Travere Therapeutics sales representatives will rapidly cover the 60 key centers to inform them of the availability of Bylvay for the treatment of pruritus in PFIC and patient access services.

Once Bylvay is prescribed, HCPs and families will have the option to use Albireo Assist™, which is a customized patient support program built with input from patient advocates.

The program features dedicated, US-based regional Care Coordinators employed by Albireo who will investigate benefits and review financial assistance options to help ensure optimal patient access.

They will also proactively assist with facilitating dosing changes, lab work, refill reminders, reauthorization and other activities.

Bylvay is the first commercially available drug for Albireo in the United States. With the U.S. approval, the FDA issued a Rare Pediatric Disease Priority Review Voucher (PRV), which the Company plans to monetize.

The Company had cash and cash equivalents of $186.3 million as of June 30, 2021 (unaudited) and anticipates the 2021 operating cash burn will be between $130-$135 million.

Excluding any proceeds from the planned sale of the PRV, the Company believes that its cash and cash equivalents will fund its operating expenses and capital expenditure requirements into 2023, which should be sufficient to launch Bylvay and expansion beyond PFIC.

Albireo is also studying the use of Bylvay in other rare pediatric cholestatic liver diseases with the BOLD Phase 3 clinical trial in patients with biliary atresia and the ASSERT Phase 3 clinical trial in Alagille syndrome.

Topline data from the ASSERT trial is expected in 2022 and the BOLD trial expected in 2024.”

https://ir.albireopharma.com/news-releases/news-release-details/multimedia-update-albireo-announces-fda-approval-bylvaytm

Vaccine inequity undermining global economic recovery

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Vaccine inequity undermining global economic recovery

July 22, 2021: “New Global Dashboard on COVID-19 Vaccine Equity finds low-income countries would add $38 billion to their GDP forecast for 2021 if they had the same vaccination rate as high-income countries.

Global economic recovery at risk if vaccines are not equitably manufactured, scaled up and distributed.  

COVID-19 vaccine inequity will have a lasting and profound impact on socio-economic recovery in low- and lower-middle income countries without urgent action to boost supply and assure equitable access for every country, including through dose sharing, according to new data released today by the United Nations Development Programme (UNDP), the World Health Organization (WHO) and the University of Oxford.

An acceleration in scaling up manufacturing and sharing enough vaccine doses with low-income countries could have added $38 billion to their GDP forecast for 2021 if they had similar vaccination rates as high income countries.

At a time when richer countries have paid trillions in stimulus to prop up flagging economies, now is the moment to ensure vaccine doses are shared quickly, all barriers to increasing vaccine manufacturing are removed and financing support is secured so vaccines are distributed equitably and a truly global economic recovery can take place.

A high price per COVID-19 vaccine dose relative to other vaccines and delivery costs – including for the health workforce surge – could put a huge strain on fragile health systems and undermine routine immunization and essential health services and could cause alarming spikes in measles, pneumonia and diarrhea.

There is also a clear risk in terms of foregone opportunities for the expansion of other immunization services, for example the safe and effective rollout of HPV vaccines.

Lower income countries need timely access to sustainably priced vaccines and timely financial support.

These insights come from the Global Dashboard for COVID-19 Vaccine Equity, a joint initiative from UNDP, WHO and the University of Oxford’s Blavatnik School of Government, which combines the latest information on COVID-19 vaccination with the most recent socio-economic data to illustrate why accelerating vaccine equity is not only critical to saving lives but also to driving a faster and fairer recovery from the pandemic with benefits for all.

“In some low- and middle-income countries, less than 1 per cent of the population is vaccinated – this is contributing to a two-track recovery from the COVID-19 pandemic”, said UNDP Administrator, Achim Steiner. 

“It’s time for swift, collective action – this new COVID-19 Vaccine Equity Dashboard will provide Governments, policymakers and international organisations with unique insights to accelerate the global delivery of vaccines and mitigate the devastating socio-economic impacts of the pandemic.”

According to the new Dashboard, which builds on data from multiple entities including the IMF, World Bank, UNICEF and Gavi, and analysis on per capita GDP growth rates from the World Economic Outlook, richer countries are projected to vaccinate quicker and recover economically quicker from COVID-19, while poorer countries haven’t even been able to vaccinate their health workers and most at-risk population and may not achieve pre-COVID-19 levels of growth until 2024.

Meanwhile, Delta and other variants are driving some countries to reinstate strict public health social measures.

This is further worsening the social, economic and health impact, especially for the most vulnerable and marginalised people. Vaccine inequity threatens all countries and risks reversing hard won progress on the Sustainable Development Goals.

“Vaccine inequity is the world’s biggest obstacle to ending this pandemic and recovering from COVID-19,” said Dr Tedros Adhanom Ghebreyesus, Director-General of the World Health Organization.

“Economically, epidemiologically and morally, it is in all countries’ best interest to use the latest available data to make lifesaving vaccines available to all.”

Designed to empower policy makers and development partners to take urgent action to reduce vaccine inequity, the Global Dashboard breaks down the impact of accessibility against a target for countries to vaccinate their at-risk populations first to reduce mortality and protect the health system and then move on to vaccinating larger shares of the population to reduce disease burden and re-open socio-economic activity.

The Dashboard is facilitated by the Global Action Plan for Healthy Lives and Well-being for All (SDG3 GAP), which aims to improve collaboration across the multilateral system to support an equitable and resilient recovery from the pandemic and drive progress towards the health-related SDGs.”

https://www.who.int/news/item/22-07-2021-vaccine-inequity-undermining-global-economic-recovery

AstraZeneca completed the acquisition of Alexion Pharmaceuticals

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AstraZeneca completed the acquisition of Alexion Pharmaceuticals

July 21, 2021: “AstraZeneca completed the acquisition of Alexion Pharmaceuticals, Inc. (Alexion).

The closing of the acquisition marks the Company’s entry into medicines for rare diseases and the beginning of a new chapter for AstraZeneca.

AstraZeneca now has an enhanced scientific presence in immunology and, through Alexion’s innovative complement-biology platform and robust pipeline, will continue to pioneer the discovery and development of medicines for patients with rare diseases.

Rare diseases represent a significant unmet medical need and becomes a high-growth opportunity for the Company.

Pascal Soriot, Chief Executive Officer, said: “Today we welcome our new colleagues from Alexion to AstraZeneca and begin a new chapter that will augment our growth for years to come.

Our sustained R&D investment in oncology, cardiovascular and renal, as well as respiratory and immunology, has powered AstraZeneca’s transformation and now we add rare diseases, where fewer approved treatment options exist.”

Marc Dunoyer, incoming Chief Executive Officer, Alexion and Chief Financial Officer, AstraZeneca, said: “I am delighted to be working alongside my new colleagues at Alexion where we will continue to discover, develop and deliver medicines that change the lives of people suffering from rare diseases.

We look forward to also applying Alexion’s complement-biology platform across areas of AstraZeneca’s broader early stage pipeline and, significantly, to the extraordinary opportunity to extend existing and future rare disease medicines to patients in many countries where AstraZeneca already has a strong presence.”

Financial considerations
The total consideration paid to the Alexion shareholders was approximately $13.3bn in cash and 236,321,411 new AstraZeneca shares (approximately 94% of which will be represented by new AstraZeneca American Depositary Shares (ADSs)).

Admission of the new AstraZeneca shares issued to Alexion shareholders to listing on the premium listing segment of the official list of the Financial Conduct Authority (FCA) and to the trading on the London Stock Exchange’s main market for listed securities has been approved and will be effective at 08:00 BST on 22 July 2021.

Trading of the new AstraZeneca shares on Nasdaq Stockholm is expected to commence on 22 July 2021. Trading of the new AstraZeneca ADSs on the Nasdaq Stock Market will commence at 09:30 EDT on 22 July 2021.

The Alexion shares will be delisted from the Nasdaq Stock Market and Alexion will terminate its registration under the U.S. Securities Exchange Act of 1934 as soon as practicable following completion of the acquisition.

As a result of completion, the issued share capital of AstraZeneca PLC with voting rights is 1,549,116,129 ordinary shares of $0.25. No shares are held in Treasury. Therefore, the total number of voting rights in AstraZeneca PLC is 1,549,116,129.

The above figure for the total number of voting rights may be used by shareholders as the denominator for the calculations by which they will determine if they are required to notify their interest in, or a change to their interest in, AstraZeneca PLC under the FCA’s Disclosure and Transparency Rules.

AstraZeneca anticipates providing updated 2021 financial guidance for the new, combined entity in due course.

Consolidation of Alexion will start from the closing of the transaction and the first quarter of consolidated financial reporting is expected to be the third quarter of 2021 due for announcement on Friday 12 November 2021.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/acquisition-of-alexion-completed.html

FDA grants Breakthrough Therapy Designation for Venclexta in combination with azacitidine for myelodysplastic syndromes

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FDA grants Breakthrough Therapy Designation for Venclexta in combination with azacitidine for myelodysplastic syndromes

July 21, 2021: Roche announced that Venclexta® (venetoclax) in combination with azacitidine has been granted Breakthrough Therapy Designation by the U.S.FDA for the treatment of adult patients with previously untreated intermediate, high- and very high-risk myelodysplastic syndromes (MDS) based on the revised International Prognostic Scoring System (IPSS-R).

MDS are a rare group of blood cancers that gradually affect the ability of the bone marrow to produce normal blood cells.

This can lead to weakness, frequent infections, anaemia and debilitating fatigue. In some cases, MDS can also progress into acute myeloid leukaemia (AML).

Every year in the US, approximately 10,000 people are diagnosed with MDS, and the median survival for those with higher-risk MDS is approximately 18 months.

“Higher-risk MDS is associated with poor prognosis, reduced quality of life, and limited treatment options,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development.

“We are pleased that the FDA has granted Venclexta its sixth Breakthrough Therapy Designation in recognition of its potential to improve outcomes for people with MDS in combination with azacitidine.”

This designation was granted based on interim results from the phase Ib M15-531 study investigating Venclexta/Venclyxto plus azacitidine in people with previously untreated, higher-risk MDS. BTD is designed to accelerate the development and review of medicines intended to treat serious or life-threatening conditions with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies.

This is the 38th BTD for Roche’s portfolio of medicines, and the 11th designation for its haematology portfolio.

This most recent designation reinforces the potential of Venclexta/Venclyxto-based combinations across several blood cancers, including MDS.

In the US, Venclexta has been granted six BTDs by the FDA: one for previously untreated chronic lymphocytic leukaemia (CLL), two for relapsed or refractory CLL, two for previously untreated AML, and one for MDS.

Venclexta/Venclyxto is already approved in the US (as Venclexta) in combination with azacitidine, decitabine or low-dose cytarabine for the treatment of newly diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, and in the EU (as Venclyxto) in combination with hypomethylating agents, azacitidine and decitabine, for the treatment of adult patients with newly diagnosed AML who are ineligible for intensive chemotherapy.

\Venclexta/Venclyxto is also approved in the US and EU in combination with MabThera®/Rituxan® (rituximab) for the treatment of adult patients with CLL who have received at least one prior therapy; in combination with Gazyva®/Gazyvaro® (obinutuzumab) for the treatment of adult patients with previously untreated CLL; and as a monotherapy for the treatment of CLL in the presence of 17p deletion or TP53 mutation in people who are unsuitable for or have failed a B-cell receptor pathway inhibitor.

Venclexta/Venclyxto is being developed by AbbVie and Roche.

It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the US, under the brand name Venclexta, and commercialised by AbbVie outside of the US.

Related News: Roche announces efficacy of Venclexta/Venclyxto in chronic lymphocytic leukaemia

AbbVie’s VENCLEXTA® Receives FDA Approval for Acute Myeloid Leukemia

About myelodysplastic syndrome (MDS)
MDS are a rare group of blood cancers that gradually affect the ability of the bone marrow to produce normal blood cells. This can lead to weakness, frequent infections, anaemia and debilitating fatigue.

In some cases, MDS can also progress into acute myeloid leukaemia (AML).

Every year in the US, approximately 10,000 people are diagnosed with MDS and the median survival for those with higher-risk MDS is approximately 18 months.

There are several classifications of MDS – very low-risk to very high-risk – determined by the composition of the bone marrow, blood cell counts, and chromosomal alterations.

Higher-risk disease is defined as intermediate, high- or very high-risk based on the revised International Prognostic Scoring System (IPSS-R), which is a risk assessment scale that uses five prognostic indicators to predict the course of a patient’s disease.

Approximately half (45%) of patients present with higher-risk MDS, which is associated with a poorer prognosis and short life expectancy.

https://www.roche.com/media/releases/med-cor-2021-07-21.htm

Coronavirus (COVID-19) Update: July 20, 2021

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Coronavirus (COVID-19) Update: July 20, 2021

July 20, 2021: “The U.S. FDA announced the following actions taken in its ongoing response effort to the COVID-19 pandemic:

  • Acting FDA Commissioner Janet Woodcock, M.D. testified before the U.S. Senate Committee on Health, Education, Labor and Pensions in a hearing titled, The Path Forward: A Federal Perspective on the COVID-19 Response.
  • This week, at the FDA SBIA REdI Annual Conference, FDA leaders reflected on the FDA’s use of Emergency Use Authorizations (EUAs) and other resources in making drug, device and biological products available to support the public health response to the COVID-19 pandemic.

    Day 1 External Link Disclaimer and Day 2 External Link Disclaimer recordings are now available on YouTube.

    The conference continues through Friday, with additional sessions External Link Disclaimer on devices and biologics.
  • Last week, the FDA held a stakeholder call to discuss COVID-19 vaccines, including preliminary reports of Guillain-Barré Syndrome following Janssen COVID-19 vaccination. 

    Here External Link Disclaimer is an excerpt from that call where Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research discussed the importance of getting vaccinated.
  • An FDA Consumer Update, Learn More About COVID-19 Vaccines From the FDA, is now available in five additional languages: Spanish, Chinese, Korean, Tagalog, and Vietnamese. See the evidence for each COVID-19 vaccine and the reasoning behind the FDA’s Emergency Use Authorizations (EUAs).
  • Testing updates: 
    • As of today, 396 tests and sample collection devices are authorized by the FDA under emergency use authorizations (EUAs). These include 281 molecular tests and sample collection devices, 85 antibody and other immune response tests and 30 antigen tests. There are 52 molecular authorizations and one antibody authorization that can be used with home-collected samples. There is one molecular prescription at-home test, three antigen prescription at-home tests, five antigen over-the-counter (OTC) at-home tests and two molecular OTC at-home tests.
    • The FDA has authorized 11 antigen tests and seven molecular tests for serial screening programs. The FDA has also authorized 576 revisions to EUA authorizations.”

      https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-july-20-2021

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